In 1983, the US Congress passed the Orphan Drug Act (ODA) that offers financial incentives for developing drugs for rare diseases that have limited, if any, approved therapeutics. A new study published in the Orphanet Journal of Rare Diseases explores the impact that the ODA has had on the field of rare diseases over the past 40 years.
Orphan drug development in the US and EU languished for years due to high development costs and low sales until it was bolstered by legislative action. While this has brought powerful therapeutics to patients, these drugs are still prohibitively expensive. In a new Value in Health: Regional Issues article, learn about the challenges of developing generic orphan drugs and the value they could bring to patients and drugmakers.
The US Food and Drug Administration (FDA) has approved Krystal Biotech’s Vyjuvek, the first gene therapy for dystrophic epidermolyis bullosa (DEB), a rare disease that leaves affected patients, dubbed “butterfly children” with skin that damages extremely easily and heals poorly. The treatment, which would cost $24,250 per dose, is applied to wounded skin, delivering a collogen gene that is missing in patients.
Orphan drugs, treatments for rare diseases with limited therapeutic options, are usually less profitable for pharma companies due to high development costs and small patient populations. However, orphan drug designation (ODD) offers monetary incentives to companies developing drugs for rare diseases. As a result, ODD plays a role in pharma drug pricing decision-making.
Families of patients have often led funding for rare disease therapeutic development through donation campaigns. However, many drugmakers have put these programs on hold, with 26 rare disease projects shelved in the past 16 months. This has caused an outcry from patient advocates. Pharma analysts argue financial reasons are to blame.
The US Food and Drug Administration (FDA) has set a date to decide on Ipsen’s new drug application for its drug candidate palovarotene in the treatment of fibrodysplasia ossificans progressive (FOP), a rare disorder where extra-skeletal tissues like muscles and tendons are converted into bone tissue. This would be the first therapy for the disabling, progressive condition.
Director Peter Marks of the US Food and Drug Administration’s (FDA’s) Center for Biologics Evaluation and Research has announced the next stage of Operation Warp Speed, a program intended to find cures and treatments for rare diseases. The program will support drugmakers developing drugs for rare diseases and will establish close lines of communication between companies and the agency to expedite the process.
The drugmaker Sanofi announced that patients with Pompe disease, a rare disease that progressively damages the heart and muscles, will now have access to its long-term enzyme replacement therapy through the NHS. The drug was markedly more effective than the standard of care in preserving respiratory function than the current standard of care.
Specialty medications currently lead the charts in terms of new drug approvals by the US Food and Drug Administration (FDA). On top of the heap are orphan drugs, treatments for rare diseases or those with no current therapeutics. In a new Pharmacy Practice News article, learn more about the orphan drugs in the approval pipeline for 2023.
The UK’s Innovative Medicines Fund (IMF) was started in collaboration with the NHS England and National Institute for Health and Care Excellence (NICE) to provide novel medicines with unresolved uncertainties to patients with rare non-cancer diseases that have little treatment options. In doing so, the program is intended to incentivized development of drugs for rare diseases, or orphan drugs.
The US Food and Drug Administration (FDA) is maintaining silence after losing a case last year over exclusivity of an orphan drug. In the case, and appeals court ruled the agency was wrong in approving a rare disease drug that shared its main ingredient with an existing orphan drug with exclusivity. As the silence continues, industry leaders worry about orphan drug exclusivity going forward, especially with the impending drug pricing reform from the Inflation Reduction Act.